Effects of gene replacement therapy with resamirigene bilparvovec (AT132) on skeletal muscle pathology in X-linked myotubular myopathy: results from a substudy of the ASPIRO open-label clinical trial
Titel:
Effects of gene replacement therapy with resamirigene bilparvovec (AT132) on skeletal muscle pathology in X-linked myotubular myopathy: results from a substudy of the ASPIRO open-label clinical trial
Auteur:
Lawlor, Michael W. Schoser, Benedikt Margeta, Marta Sewry, Caroline A. Jones, Karra A. Shieh, Perry B. Kuntz, Nancy L. Smith, Barbara K. Dowling, James J. Müller-Felber, Wolfgang Bönnemann, Carsten G. Seferian, Andreea M. Blaschek, Astrid Neuhaus, Sarah Foley, A. Reghan Saade, Dimah N. Tsuchiya, Etsuko Qasim, Ummulwara R. Beatka, Margaret Prom, Mariah J. Ott, Emily Danielson, Susan Krakau, Paul Kumar, Suresh N. Meng, Hui Vanden Avond, Mark Wells, Clive Gordish-Dressman, Heather Beggs, Alan H. Christensen, Sarah Conner, Edward James, Emma S. Lee, Jun Sadhu, Chanchal Miller, Weston Sepulveda, Bryan Varfaj, Fatbardha Prasad, Suyash Rico, Salvador